Appropriately, it was Jack Valenti, prominent former president of the Motion Pictures Association of America, who recommended to politicians that every speech should include the six words: “let me tell you a story.” Stories have impact in ways reports do not. Eddie Redmayne as Stephen Hawking, a theoretical physicist diagnosed with a form of the motor-neuron disease amyotrophic lateral sclerosis (ALS), in The Theory of Everything, and Julianne Moore as Dr. Alice Howland, a fictional linguistics professor diagnosed with early onset Alzheimer’s, in Still Alice, were Academy Award winners last Sunday evening. These films grappled with devastating diagnoses for the patient and their loved ones, putting a face to the 30,000 Americans living with ALS and the more than 5 million Americans living with Alzheimer’s.

A less high profile but impressively high impact group of advocates for rare disease research traveled to D.C. from around the nation to tell their own – personal and nonfictional – stories about the toll visited on patients and families by a wide range of diseases that also call out for more research. Having spoken with the group early yesterday morning before they fanned out on Capitol Hill, I can attest to how well-rehearsed and determined they were to make their case. I recalled that it is patients and their families who have, historically, so often made the breakthrough difference in advocacy for research, going back to the key role of the March of Dimes in focusing the nation on the imperative of putting research to work to defeat polio, through the paradigm shifters called AIDS activists and women’s health research advocates, and many more. Now is the time for more stories to be told on Capitol Hill, at this moment of opportunity for galvanizing Congress’ increasing interest into action. Continue reading →

New Poll Data Summary booklet reveals concerns among Americans about the pace of medical progress

Majorities across the political spectrum say it is important that the new 114th Congress takes action on assuring the discovery, development and delivery of treatments and cures for diseases in the first 100 days of the legislative session (75% Democrats, 64% Republicans and 60% Independents), according to America Speaks, Volume 15, a compilation of public opinion polls commissioned by Research!America. As Congress considers numerous proposals in support of research, including the 21st Century Cures draft legislation aimed at speeding the delivery of lifesaving treatments to patients, it is notable to see public support in favor of accelerating medical progress.

“The new Congress has the opportunity to reinvigorate our research ecosystem and enact policies that will enable the private sector to expand innovation,” said Research!America Chair John Edward Porter. “Congress must work in a bipartisan fashion to realize the potential of promising studies to prevent and treat disease.”

An increasing percentage of Americans say the U.S. Food and Drug Administration (FDA) should move more quickly in order to get new treatments to patients, even if it means there may be risks. In 2015, 38% favor faster regulatory review, compared to 30% in 2013. Meanwhile, 25% say the FDA should act more slowly in order to reduce risk, even if it means patients may wait longer for treatments. Another 19% are undecided on this question and 18% do not agree with either position. Continue reading →

The cover story of this month’s National Geographic describes the recent wave of science doubt as a “pop culture meme,” featuring in-the-news examples like climate change and vaccines, and discussion of tough challenges like replicability of research, scientific literacy (of note: increased science literacy has been shown to lead to increased polarization of opinion about science), and what is meant, anyway, by effective “science communication”? The article doesn’t mention what I often call the “invisibility” problem (see, for example, data showing low percentages of Americans who can name a living scientist), but that topic was addressed directly and indirectly in several sessions at last week’s annual meeting of the American Association for the Advancement of Science (AAAS).

Data from a Pew Research poll of AAAS members show that a majority of scientists now believe that it is important to engage with the public, with a high percentage saying they do so regularly. That is welcome news. Another AAAS session brought out the importance of the quality of that engagement, exploring connecting with non-scientists in ways that is positive for both scientist and non-scientist. And, Professor Susan Fiske of Princeton spoke to an overflow crowd in her featured session about work showing that all of us – people in general – for better or worse, and with consequences to match – make quick judgments about others’ intent and their degree of competency. (Perception of competency + perception of good intent = trust.) Fiske noted that politicians are almost never trusted, although they are sometimes viewed as competent. Scientists are mostly considered competent, but they are also considered to be cold, a judgment that can throw their intentions into question. Fiske said that it is possible to change perceptions about scientists if they convey warmth and motivation to cooperate, showing ‘worthy intent.’ (If you have followed Research!America’s work in communicating to the non-science trained public, you know that we advocate saying and conveying, “I work for you.” That advice fits right in here.) Continue reading →

Today, the House Ways and Means Committee considered legislation (HR 880) to make the R&D tax credit permanent. A simplified, permanent R&D tax credit would amplify the credit’s proven positive impact on the pace of innovation and the strength of our economy. Read our statement submitted for the record here. We know from recent polling that Americans value the R&D tax credit, even if the federal government loses some revenue annually. This and more poll data will be highlighted in our 2015 Poll Data Summary next week, so keep an eye out for this timely read!

Tax reform is one component of the policy climate in which R&D flourishes…or doesn’t. Dr. Mark McClellan, Research!America Board member and Director of the Health Care Innovation and Value Initiative at Brookings, has co-authored two relevant articles in the February issue of Health Affairs: one that looks at ways to bolster the inadequate pipeline of high priority antibiotics as resistant strains of infectious illness proliferate; and a second article that discusses approaches to assessing the “innovativeness” of medical advances. The significance of the former is self-evident. The significance of the latter is multi-faceted, but one important reason to gauge “innovativeness” is to help answer the value question central to reimbursement decisions and other policies. There is no easy way to assign the “right” value to products that could have broad-scale, tangible and intangible, short- and long-term impact on our health, wellbeing and productivity, the economy, federal, state and local budgets, national security, and more. This is an important undertaking, since if we don’t understand exactly where medical progress falls among our national and individual priorities, we are bound to under- or over-incentivize it. And that’s costly on many, many levels. Both of the studies Mark contributed to are well worth the read. Continue reading →

By Martine Rothblatt, PhD, Chairman and CEO of United Therapeutics Corporation.

Founded in 1996, United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of unique products to address the unmet medical needs of patients with chronic and life-threatening conditions. We have four approved products on the market today and we are not stopping there! From the United States to Europe to the Asia Pacific, we are proud of our multicultural business environment where employees can collaborate with people all over the world. As a group, we are relentless in our pursuit of “medicines for life”® and continue our research into treatments for pulmonary arterial hypertension, cancer, and some of the world’s most complicated viral illnesses.

We are proud to partner with Research!America to promote better medical advancements, biomedical research, and overall greater global health initiatives. We have seen first-hand how tireless research and dedication to a cause can change the lives of thousands of patients and their loved ones. We began our story by conducting extensive research on a treatment for a deadly disease so rare, Pulmonary Arterial Hypertension (PAH), that other medical companies had abandoned any pursuits for treatments or a cure. Continue reading →

Today is the 13th anniversary of the American Dental Association‘s Give Kids a Smile Day program. Aimed at raising awareness and helping to address the high level of oral disease in kids, especially in underserved communities, this program enables volunteers to provide free dental care to those in need and urges policymakers to increase funding for children’s oral health.

Dental caries, referred to as a “silent epidemic” by former U.S. Surgeon General David Satcher, are the cause of many otherwise preventable health issues. Investing in comprehensive oral health care for children will result in fewer instances of dental caries in adulthood. Studies have shown that increased funding for combating oral health conditions results in fewer emergency room visits and lower health care costs across the board.

“Addressing preventable disease is the norm in today’s health care system. It is the most effective way to reduce costs and improve health. This is no different for oral disease. Working together, industry and its partners can enhance the prevention and treatment of oral disease through the development and testing of new treatments that will improve oral health, overall health and quality of life for all.” – Dr. Foti Panagakos, DMD, Ph.D, global director of scientific affairs, Colgate-Palmolive

The President’s FY16 budget was released this week. It makes the case for, and does its math on the basis of, the end of sequestration. Hallelujah! If Congress agrees to discard this monster that no one wanted to begin with, perhaps the executive and legislative branches can work to position our nation for a better future. That most fundamental of goals has been neglected for far too long and it is now time to make strategic investments for our nation.

In that context, we’re excited about the level of interest in medical progress that has in many ways defined the early days of the 114th Congress. There are several bills in play that would recalibrate research and development policies and research funding to achieve a pace that meets the needs of patients and secures the public’s safety. We believe that NIH, CDC, AHRQ, FDA, and NSF should be the focus of a major strategic investment this year so that we can accelerate medical progress, so that we can put the brakes on Alzheimer’s, bolster our anti-infectious disease arsenal, and meet the other massive health/economic/fiscal/national security challenges before us. Read more here.

Dr. Margaret Hamburg has announced that she will be stepping down from her post as FDA Commissioner. She is an exemplary leader who has fulfilled a crucial role for the nation with characteristic vision, dedication and skill. More here.

And speaking of FDA, now is the time to share your input on the 21st Century Cures initiative. I have had the privilege of weighing in with Reps. Upton (R-MI) and DeGette (D-CO) and their staff members, and I encourage you to do the same. Join the conversation on social media with the hashtag #Cures2015 and send formal comments to cures@mail.house.gov. Do it now: a new draft could come out as soon as two weeks from today. As I mentioned last week, Senator Alexander (R-TN) has launched an effort similar to 21st Century Cures. When details become available, Research!America will update you on ways to contribute to the Senate process.

As you strategize what your contributions to the various initiatives, working groups and legislative proposals will be, you might find it helpful to huddle with other organizations in the field. We’re planning a stakeholder strategy meeting for Research!America members, hosted at the offices of the Society for Neuroscience. Join us next Wednesday Feb. 11 at 1pm. Space is limited, so please reserve a seat by e-mailing Jordan Gates at jgates@researchamerica.org.

Exciting news Tuesday from Research!America board member and former Representative Patrick J. Kennedy and former Surgeon General Dr. David Satcher. Their partnership to address the state of mental health and addiction in this country and to achieve health equity is now formalized as The Kennedy Center for Mental Health Policy and Research within the Satcher Health Leadership Institute at the Morehouse School of Medicine in Atlanta. None of us should settle for the status quo approach when it comes to achieving mental health. It was noted that the military sees mental health explicitly as a “force multiplier;” giving our nation a competitive edge. I see the Kennedy-Satcher partnership as a force multiplier, too – there are no two people more passionate and none with a stronger track record as a leader for mental health. See key findings from a poll they commissioned here.

Mark your calendars, tomorrow (Friday Feb. 6) is an important day! It is “Give Kids a Smile Day” which promotes the importance of oral health and provides dental care to underserved children. In support of this effort, we are releasing our new Children’s Dental Health Research fact sheet. And speaking of David Satcher, he made dental health one of the foci of his tenure as Surgeon General. It was Dr. Satcher who called oral disease the “silent epidemic.” It is truly important to understand that children’s dental health is not a luxury; it is core to their health and wellbeing.

Tomorrow is also the last call for scientists at the post-graduate level and above to submit applications for “Connecting the Dots: Effectively Communicating Science to Non-Scientists,” a two-day program that we are hosting in partnership with the George Washington University School of Media and Public Affairs. Submit your application here by Friday.

We are pleased that the President’s FY16 budget proposal calls for the elimination of sequestration and starts an overdue conversation about better aligning resources for public health and medical progress, given their importance to the American people and to the health of our economy. It is critical that we ramp up initiatives that focus on precision medicine, Alzheimer’s, antimicrobial resistance and other growing health threats, but these investments should supplement, not supplant, the imperative of making up for a decade’s worth of lost ground. We believe that Congress and the White House can, and must, unify behind the vision encapsulated in the bipartisan Accelerating Biomedical Research Act. Medical progress is not just a health imperative, it is a strategic imperative, integral to national security, fiscal stability and economic progress. Leaders on both sides of the aisle clearly appreciate that the time is now to turn ideas into reality. It may be a truism, but where there’s a will, there’s a way. Continue reading →

President Obama’s Precision Medicine Initiative could potentially drive medical and health research into exciting, new territory as we advance efforts to develop the right treatments at the right time for individual patients. A laser-focus approach that takes into account a patient’s genetic profile, environment and lifestyle is critical to treat diseases such as cancer which afflicts millions of Americans. Only about a quarter of Americans believe the U.S. has the best health care system in the world, according to public opinion polling commissioned by Research!America. This initiative could help reverse both the perception and the reality with targeted treatments that will save lives and improve health care delivery.

This initiative is an important development for patients, physicians and researchers who will benefit from a stronger national commitment to precision medicine and for those who may yet take advantage of the new tools and therapies that will result from this effort. And many Americans are ready to support this endeavor. Polls show more than half say they are willing to share their personal health information to advance research and help improve patient care, and a majority believe that elected officials should listen to advice from scientists. This initiative is a major step towards building a stronger public-private partnership to leverage health data and technology to accelerate the discovery and development of tailored treatments for patients. Continue reading →

I warn you that today’s letter is long. There is a lot going on; suddenly, lots of people in Congress are staking out leadership roles as champions for research! On Tuesday, the first draft of the much anticipated 21st Century Cures legislation was released. Chairman Fred Upton (R-MI-06) and Congresswoman Diana DeGette (D-CO-01), who will jointly receive Research!America’s 2015 Edwin C. Whitehead Award for Medical Research Advocacy at our Advocacy Awards dinner on Wednesday, March 11 (click here to join us for the event), have been partnering on this effort since last spring. Congresswoman DeGette has not endorsed the current draft, but she has made it clear that she continues to be committed to the process. We are, too. Over the coming weeks, we will be meeting with the 21st Century Cures team and participating in a variety of discussions on the intricacies of the bill.

Among our priorities will be to ensure that basic discovery is not neglected (a house built on sand…), and to make sure that, for the U.S. Food and Drug Administration (FDA) and other agencies – already struggling to fulfill their current responsibilities – if given new “to do” lists, must also be given new resources. We will continue to push for final bipartisan language that effectively boosts the return on medical progress by accelerating discovery, development and delivery. See our statement on the 21st Century Cures release here. And click here to see the Committee’s documents. They have invited suggestions on the draft, and ask that you send your comments to cures@mail.house.gov. Continue reading →

The release of the 21st Century Cures Initiative discussion draft is a major bipartisan accomplishment that represents a truly remarkable diversity of innovative ideas to speed the delivery of lifesaving treatments to patients – a testament to the extraordinary commitment of Congressman Fred Upton (R-MI), Congresswoman Diana DeGette (D-CO) and their respective staff members. The Initiative could be a game changer for the medical innovation ecosystem with provisions touching on virtually all phases of the research and development pipeline – from basic and applied research, to FDA review, to coverage and access. Among the many beneficial provisions Research!America fought for is a measure to reduce the administrative burden on researchers. We look forward to working with the 21st Century Cures team to greatly boost our nation’s commitment to groundbreaking research and drug development.

The State of the Union (SOTU) message is the annual opportunity for the President to outline his goals. Read my statement on it here. The SOTU historically provides a platform for the executive and legislative branches to identify commonalities, or sharpen differences. Topics in our sights on which Congress and the Administration can work together should they choose (meaning if their constituents demand it!) include innovation, research and development, and 21st century business success. In his speech and in a more detailed proposal, the President calls for a significant increase in funding for antibiotic discovery, Alzheimer’s research, the BRAIN Initiative and precision medicine. We urge the President and Congress to go further, working together to advance a strategic “moonshot” that re-energizes our national commitment to science, very much including basic science. Basic discovery is truly the foundation for all of our nation’s scientific advances. It’s pretty simple. As Dr. Roger Perlmutter, Executive Vice President at Merck, said this week in The New York Times: “Since we don’t know how the machine [the human body] works, we don’t know what to do when it breaks.”

Investment in basic and clinical research isn’t a “nice to have” proposition; it’s essential, leading as nothing else will, to good news for patients. An op-ed this week from Executive Vice Chancellor for Medical Affairs at the Washington University School of Medicine in St. Louis and Research!America board member Dr. Larry Shapiro discusses the university’s work, funded by the federal government, which has led to advances in early diagnosis of Alzheimer’s before symptoms are perceptible. Continue reading →

In his State of the Union address, President Obama highlighted the important role of research and innovation in growing a more prosperous and healthier nation. We’re excited about the launch of the Precision Medicine Initiative, which comes at a time when the challenge of conquering disease – all along the research spectrum, from discovery to translation to innovation and application – has never been more within our grasp. The inspiring story of William Elder, Jr. a medical school student and cystic fibrosis survivor, shows that science can deliver breakthroughs for patients with cystic fibrosis, cancer, Alzheimer’s and other diseases. The remarkable ability of our nation’s researchers to advance precision medicine to hone targeted treatments to improve individual patient outcomes is a compelling example of what can be accomplished with federal support. We’re getting closer and closer to achieving treatments that save time, save money and save lives because they are right the first time.

We can’t afford to ease up on our commitment to research, to assure we can put a whole range of diseases in the history books. A further reason, noted by the President, is that we need robust funding and policies to ensure we’re not behind the eight ball addressing domestic or global outbreaks like Ebola. Current funding levels for federal health agencies put researchers at an extreme disadvantage in pursuing studies that have the potential to cure disease and improve quality of life, and tax policies have stymied the development of new drugs. Policymakers must pivot from short-sighted thinking to formulating a long-term strategy that will bring new treatments across the finish line and spur growth in quality jobs. We think it’s past time to adopt a national strategy that will assure the U.S. retains its world leadership in science and innovation. A new Blue Ribbon Commission established by Congress to explore how science is perceived by the public will help stimulate a meaningful conversation with Americans about the societal and economic benefits of science. Continue reading →

I start this letter by sharing our excitement that Congressman Fred Upton (R-MI-06) and Congresswoman Diana DeGette (D-CO-01) will receive the Edwin C. Whitehead Award for Medical Research Advocacy this year. Their leadership on the 21st Century Cures initiative is just the latest example of their “all-in” commitment to medical progress. The (loose) theme of this week’s letter is “walk the talk”: there are few leaders in Congress who have more consistently or productively adhered to that mantra. Read our press release here.

Our Whitehead winners are shepherding a change in direction for public policy related to medical innovation. Complacency and neglect are out and bold action to bolster resources and achieve time- and cost-saving efficiencies is in. Not a moment too soon: a report authored by Dr. Hamilton Moses et. al. this week in the Journal of the American Medical Association (JAMA) documents the disturbing slowdown in U.S. investment in medical research, made all the more striking in contrast to dramatically increased investment by other nations. The authors emphasize that languishing investments cannot and will not produce the meaningful medical breakthroughs our society is expecting. Continue reading →

By Caleph B. Wilson, Ph.D., a biomedical researcher at the University of Pennsylvania, logistics director of the National Science Policy Group, science communicator and STEM outreach advocate. Follow him on Twitter as @HeyDrWilson.

With the 114th Congress underway, the scientific community is looking forward to sharing new research breakthroughs and advocating for STEM during a series of congressional visits to Capitol Hill. In some instances, scientists and trainees will assist writing congressional briefs and give testimony to House and Senate committees on science, technology and health.

While Congress is considering science policy initiatives, positions and funding, there are a few things in the early-career scientist “wish list” that would make improvements and maintain the United States’ leading position in the scientific enterprise.

Throughout 2014, early-career scientists discussed specific issues in science policy groups, on social media and in articles that need to be addressed. These are some of the highlights of the conversations that have been put in a “Wish List” that hopefully Congress and policymakers will strongly consider.

National Science Foundation (NSF) and National Institutes of Health (NIH) funding that is predictable and keeps pace with inflation.

In the early 1990s, the NIH budget increased dramatically. However, over the last 10 years the NIH budget has flat-lined and even decreased at times. Unfortunately, the budget has not kept pace with inflation and rising costs of executing experiments. With changes in the economy and the sweeping budget cuts that came in with sequestration, government agencies, institutions and investigators can better plan with predictable budget appropriations that keep pace with scientific opportunity. Continue reading →